News

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…

Health Canada has approved Ruzurgi (amifampridine) for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in individuals ages 6 or older. In Canada, the oral medication will be commercialized by Médunik Canada, per an agreement with Jacobus Pharmaceuticals, the original developer of Ruzurgi.  “We are proud…

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…

Health Canada has approved the use of oral Firdapse (amifampridine) to treat people with Lambert-Eaton myasthenic syndrome (LEMS). Catalyst Pharmaceuticals‘ request for Firdapse’s approval in Canada was assigned priority review. The oral therapy (given in 10 mg tablets) is already available for…

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films to an audience.  Co-founders Daniel DeFabio and Bo Bigelow, who are both fathers of children with…

A post-exercise facilitation test, which measures muscle activity following voluntary muscle contraction or electrical stimulation, can help identify the effects of carpal tunnel syndrome (CTS) on nerves and muscles in people with Lambert-Eaton myasthenic syndrome (LEMS), a case report shows. The study, “Use of Post-Exercise…

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…

A new scoring tool can be used to effectively estimate the risk of developing small cell lung cancer (SCLC) for a person with Lambert-Eaton myasthenic syndrome (LEMS), according to a new study. Smoking, being age 50 or older, and significant weight loss within three months of LEMS onset all…

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support activities that align with the foundation’s Rare Belonging focus, a set of funding priorities aimed at improving the…

People with Lambert-Eaton myasthenia syndrome (LEMS) have a specific response pattern to repetitive nerve stimulation — known as RNS — that may enable physicians to easily distinguish such cases from the neuromuscular condition myasthenia gravis (MG), a study reports. The study, “Lambert-Eaton myasthenia syndrome:…